Studies
| Study First Submitted Date | 2020-08-25 |
| Study First Posted Date | 2020-08-28 |
| Last Update Posted Date | 2021-10-20 |
| Verification Month Year | October 2021 |
| Verification Date | 2021-10-31 |
| Last Update Posted Date | 2021-10-20 |
Detailed Descriptions
| Sequence: | 20753032 |
| Description | The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis. |
Facilities
| Sequence: | 200369962 | Sequence: | 200369963 | Sequence: | 200369964 | Sequence: | 200369965 | Sequence: | 200369966 | Sequence: | 200369967 | Sequence: | 200369968 | Sequence: | 200369969 | Sequence: | 200369970 | Sequence: | 200369971 | Sequence: | 200369972 | Sequence: | 200369973 | Sequence: | 200369974 | Sequence: | 200369975 |
| Name | University of California, Los Angeles | Name | Stanford University | Name | University of California San Francisco | Name | Georgetown University | Name | Advent Health | Name | Children’s Healthcare of Atlanta/Emory University | Name | Ann & Robert H. Lurie Children’s Hospital | Name | Ochsner Clinic Foundation | Name | University of Minnesota | Name | The Children’s Hospital at Montefiore | Name | University of Oklahoma | Name | Baylor College of Medicine | Name | University of Virginia | Name | Medical College of Wisconsin |
| City | Los Angeles | City | Redwood City | City | San Francisco | City | Washington | City | Orlando | City | Atlanta | City | Chicago | City | New Orleans | City | Minneapolis | City | New York | City | Oklahoma City | City | Houston | City | Charlottesville | City | Milwaukee |
| State | California | State | California | State | California | State | District of Columbia | State | Florida | State | Georgia | State | Illinois | State | Louisiana | State | Minnesota | State | New York | State | Oklahoma | State | Texas | State | Virginia | State | Wisconsin |
| Zip | 90095 | Zip | 94063 | Zip | 94158 | Zip | 20007 | Zip | 32804 | Zip | 30329 | Zip | 60611 | Zip | 70121 | Zip | 55455 | Zip | 10467 | Zip | 73104 | Zip | 77030 | Zip | 22903 | Zip | 53226 |
| Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States | Country | United States |
Conditions
| Sequence: | 52251354 |
| Name | Alagille Syndrome |
| Downcase Name | alagille syndrome |
Id Information
| Sequence: | 40217127 |
| Id Source | org_study_id |
| Id Value | MRX-EAP |
Countries
| Sequence: | 42632048 |
| Name | United States |
| Removed | False |
Interventions
| Sequence: | 52564284 |
| Intervention Type | Drug |
| Name | maralixibat |
| Description | Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor |
Keywords
| Sequence: | 79982414 | Sequence: | 79982415 | Sequence: | 79982416 | Sequence: | 79982417 | Sequence: | 79982418 | Sequence: | 79982419 | Sequence: | 79982420 | Sequence: | 79982421 |
| Name | Liver Disease | Name | ALGS | Name | Maralixibat | Name | Bile Duct Diseases | Name | Digestive System Diseases | Name | Biliary Tract Diseases | Name | Cholestasis | Name | Pruritis |
| Downcase Name | liver disease | Downcase Name | algs | Downcase Name | maralixibat | Downcase Name | bile duct diseases | Downcase Name | digestive system diseases | Downcase Name | biliary tract diseases | Downcase Name | cholestasis | Downcase Name | pruritis |
Browse Conditions
| Sequence: | 193793462 | Sequence: | 193793463 | Sequence: | 193793464 | Sequence: | 193793465 | Sequence: | 193793466 | Sequence: | 193793467 | Sequence: | 193793468 | Sequence: | 193793469 | Sequence: | 193793470 | Sequence: | 193793471 | Sequence: | 193793472 | Sequence: | 193793473 | Sequence: | 193793474 | Sequence: | 193793475 | Sequence: | 193793476 | Sequence: | 193793477 | Sequence: | 193793478 | Sequence: | 193793479 | Sequence: | 193793480 |
| Mesh Term | Alagille Syndrome | Mesh Term | Pruritus | Mesh Term | Syndrome | Mesh Term | Disease | Mesh Term | Pathologic Processes | Mesh Term | Skin Diseases | Mesh Term | Skin Manifestations | Mesh Term | Cholestasis, Intrahepatic | Mesh Term | Cholestasis | Mesh Term | Bile Duct Diseases | Mesh Term | Biliary Tract Diseases | Mesh Term | Digestive System Diseases | Mesh Term | Liver Diseases | Mesh Term | Heart Defects, Congenital | Mesh Term | Cardiovascular Abnormalities | Mesh Term | Cardiovascular Diseases | Mesh Term | Abnormalities, Multiple | Mesh Term | Congenital Abnormalities | Mesh Term | Genetic Diseases, Inborn |
| Downcase Mesh Term | alagille syndrome | Downcase Mesh Term | pruritus | Downcase Mesh Term | syndrome | Downcase Mesh Term | disease | Downcase Mesh Term | pathologic processes | Downcase Mesh Term | skin diseases | Downcase Mesh Term | skin manifestations | Downcase Mesh Term | cholestasis, intrahepatic | Downcase Mesh Term | cholestasis | Downcase Mesh Term | bile duct diseases | Downcase Mesh Term | biliary tract diseases | Downcase Mesh Term | digestive system diseases | Downcase Mesh Term | liver diseases | Downcase Mesh Term | heart defects, congenital | Downcase Mesh Term | cardiovascular abnormalities | Downcase Mesh Term | cardiovascular diseases | Downcase Mesh Term | abnormalities, multiple | Downcase Mesh Term | congenital abnormalities | Downcase Mesh Term | genetic diseases, inborn |
| Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48394591 | Sequence: | 48394592 |
| Agency Class | INDUSTRY | Agency Class | INDUSTRY |
| Lead Or Collaborator | lead | Lead Or Collaborator | collaborator |
| Name | Mirum Pharmaceuticals, Inc. | Name | Clinigen, Inc. |
Eligibilities
| Sequence: | 30812160 |
| Gender | All |
| Minimum Age | 12 Months |
| Maximum Age | N/A |
| Criteria | Inclusion Criteria: Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC) Clinical and/or genetic diagnosis of ALGS Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program No other therapeutic options or access to any ongoing ALGS clinical trials Exclusion Criteria: Diagnosis with a cholestatic liver disease other than ALGS Female who is pregnant or breastfeeding Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program Past medical history of compensated or decompensated cirrhosis Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action |
| Adult | True |
| Child | True |
| Older Adult | True |
Calculated Values
| Sequence: | 254051726 |
| Number Of Facilities | 14 |
| Registered In Calendar Year | 2020 |
| Were Results Reported | False |
| Has Us Facility | True |
| Has Single Facility | False |
| Minimum Age Num | 12 |
| Minimum Age Unit | Months |
Links
| Sequence: | 4394351 | Sequence: | 4394352 | Sequence: | 4394353 | Sequence: | 4394354 |
| Url | https://ghr.nlm.nih.gov/condition/alagille-syndrome | Url | https://clinicaltrials.gov/ct2/info/fdalinks | Url | https://mirumpharma.com | Url | https://algseap.com/ |
| Description | Genetics Home Reference – Alagille syndrome | Description | US FDA Resources | Description | Mirum Pharmaceuticals homepage | Description | Mirum webpage specific to ALGS EAP |
Responsible Parties
| Sequence: | 28924524 |
| Responsible Party Type | Sponsor |